BPC September 05 update

Global Blood Therapeutics GBT receives priority review for sickle cell disease NDA - shares close up 11%

Price and Volume Movers

Bristol-Myers Squibb Company (NYSE: BMY) announced that its Phase 3 CheckMate 548 trial evaluating Opdivo and the current standard of care, versus the standard of care alone, did not meet the primary endpoint of progression-free survival (PFS), in patients with newly diagnosed glioblastoma multiforme (GBM) patients. The trial will continue to allow the other primary endpoint, overall survival (OS), to mature.

Global Blood Therapeutics, Inc. (NASDAQ: GBT) shares closed up 11% to $52.66 following news the FDA has accepted its New Drug Application (NDA) seeking accelerated approval for voxelotor, for the treatment of sickle cell disease (SCD). The PDUFA date under priority review is February 26, 2020. The company noted that the FDA indicated that it is not currently planning to hold an advisory committee meeting.

Zafgen, Inc. (Nasdaq:ZFGN) shares closed down 14% to $0.70. The company announced that data from its in vivo animal trial to establish safety margins for ZGN-1061, is not expected to lift the clinical hold currently in place. The company intends to explore strategic options, which may include an acquisition, merger, business combination, in-licensing, or other strategic transactions.


Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume


Autolus Therapeutics Plc (AUTL): $11.93; +22%.

Novan, Inc. (NOVN): $2.50; +15%.

Liquidia Technologies, Inc. (LQDA): $3.88; +14%.

Neon Therapeutics, Inc. (NTGN): $2.91; +14%.

Karyopharm Therapeutics Inc. (KPTI): $9.45; +11%.


Mallinckrodt Public Limited Company (MNK): $1.59; -39%.

Plus Therapeutics, Inc. (PSTV): $11.81; -16%.

uniQure N.V. (QURE): $47.66; -15%.

Aeterna Zentaris Inc. (AEZS): $1.79; -9%.

Neurotrope, Inc. (NTRP): $4.77; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACRS – Aclaris Therapeutics Inc.
A-101 THWART-1 and THWART-2
Common warts (verruca vulgaris)

Phase 3 Phase 3 data met all endpoints - September 16, 2019 and October 24, 2019.
$47.3 million

ACRS – Aclaris Therapeutics Inc.
Androgenetic alopecia (AGA) - Hair loss

Phase 2 Partner required to further development.
$47.3 million

BMY – Bristol-Myers Squibb Company
CM-548 - Opdivo
First-line Glioblastoma (GBM) cancer

Phase 3 Phase 3 trial did not meet PFS endpoint. Trial to continue to allow OS endpoint to mature - September 5, 2019. Data due 2021.
$133.3 billion

ESPR – Esperion Therapeutics Inc.
Bempedoic Acid (ETC-1002) CLEAR Outcomes
Cardiovascular Diseases

Phase 3 Phase 3 completion of enrolment announced September 5, 2019. Trial to be completed in 2H 2022.
$1.4 billion

GBT – Global Blood Therapeutics Inc.
Sickle cell disease

Approved FDA Approval announced November 25, 2019.
$3.9 billion

IMUX – Immunic Inc.
Ulcerative colitis

Phase 2 Phase 2 data due 4Q 2021.
$78.5 million

RCKT – Rocket Pharmaceuticals Inc.
Leukocyte Adhesion Deficiency-I (LAD-I)

Phase 1 Phase 1 initial data released December 9, 2019 showed early evidence of safety and potential efficacy
$1.1 billion

ZFGN – Zafgen Inc.

Phase 1 Data from in vivo animal model unlikely to resolve current clinical hold - September 5, 2019.
$42.6 million